September 10, 2014
By Riley McDermid, BioSpace.com Breaking News Sr. Editor
A father who quit his job working as a fund manager on Wall Street to found a firm dedicated to curing his son’s Duchenne muscular dystrophy has just inked a deal with pharma giant Pfizer Inc. to test one of its early-stage experimental drugs.
Ilan Ganot quit his job working with hedge funds at JPMorgan in October 2012 when his son was diagnosed and quickly went into high gear founding biotech company Solid Ventures to exclusively focus on cures for Duchenne. He raised an initial $17 million round of funding for the Boston-based firm.
Joel Schneider, scientific analyst at Boston-based Solid, said the firm made the deal a few weeks ago but did not issue a formal announcement. In an interview today with CNBC, Schneider said the deal with Pfizer came about through Carl Morris, Pfizer’s senior director of the Rare Disease Research Unit in Cambridge.
Duchenne is a genetic muscle-wasting disease with no cure and no drugs are approved to treat it in the U.S."In rare diseases—and there are thousands of them—we find it difficult sometimes to substantiate the economic value, to justify for big companies to roll up their sleeves and jump right in,” Ganot told CNBC. “What we’re left with is a lot of patients that are left without drugs, and a lot of science that is not getting developed.”
Although he declined to pinpoint Pfizer’s therapeutic area for the drug, he did say Solid remains hopeful it could to move directly to the mid-stages of clinical testing because of how well scientists understand the molecular structures involved.
But the for-profit company isn’t resting on its laurels: It has formed a subsidiary focused on gene therapy called Solid GT, which “if it works changes the disease completely,” Ganot told CNBC. “It’s relevant for every patient.”
Solid GT is chaired by the Wilson, a professor and director of the University of Pennsylvania’s Gene Therapy Program. It aims to take Duchenne’s main genetic component dystrophin and introduce it to in order to produce a muscle function protein also called dystrophin.
The biotech industry has long been excited about gene therapy, which involves adding preselected genes into patients’ cells in an effort to create disease resistance. Though gains in the area have been staggered, recent years have seen companies like Sangame and Bluebird Bio meet with some limited success.
